CNGA1

MUNICH, Germany I August 22, 2023 I ViGeneron GmbH (ViGeneron), a next-generation gene therapy company, today announced that the European Medicines Agency (EMA) has approved the Clinical Trial Application (CTA) for VG901, a potentially transformative gene therapy to treat CNGA1-associated Retinitis Pigmentosa (RP), an ocular disorder currently lacking approved therapies. VG901 uses vgAAV, ViGeneron’s proprietary adeno-associated virus (AAV) vector to deliver the CNGA1 gene via intravitreal (IVT) injection, thereby reducing the risk of retinal damage, which is associated with subretinal administration. The Phase Ib dose-escalation clinical trial will evaluate safety, tolerability and efficacy of VG901 in patients with RP due to biallelic CNGA1 mutations. This trial is part of an international regulatory strategy for the clinical development of VG901. Further interactions with regulatory authorities are currently being prepared. Dr. Caroline Man Xu, Co-founder and CEO of ViGeneron said: “This is an important step in our mission to bring a novel gene therapy to patients born with CNGA1 mutations, to save their eyesight, potentially. We are excited to develop this first-in-class gene therapy to provide a potential cure for patients who currently have no treatment options.” In preclinical studies, ViGeneron observed that VG901 has the potential to supplement the CNGA1 gene in a mouse model of RP. In addition, a GLP-safety study applying a single intravitreal injection followed by a 6-month post treatment observation period confirmed the safety of VG901. Furthermore, ViGeneron’s manufacturing partner, WuXi Advanced Therapies (WuXi ATU), has successfully completed GMP manufacturing of the clinical trial material. VG901 was developed using ViGeneron’s proprietary next generation gene therapy technology platform vgAAV – a platform based on AAV2 – to be delivered by IVT. This improved vector provides a greater ease of administration and wider vector distribution compared with subretinal injection, commonly used in retinal gene therapy studies. Side by side comparison of in vivo expression versus a competitor capsid demonstrated superior IVT transduction efficiency.  In addition, IVT delivery avoids the risk of retinal damage associated with subretinal administration and is also less invasive than suprachoroidal injection. Due to its ability to cross barriers, this vector is suitable for various routes of administration and target organs. It can be combined with our second platform, which allows delivery of genes larger than 5kb, as required for some more prevalent rare diseases. About Retinitis Pigmentosa (RP) Retinitis Pigmentosa (RP) is a group of related eye disorders that cause progressive vision loss. There are currently no approved treatments for RP. RP initially presents as nighttime blindness during childhood or early adulthood, progressing to peripheral visual field loss and “tunnel vision,” central visual impairment, reduced visual acuity and, ultimately, complete blindness. Retinitis Pigmentosa is the most common type of inherited retinal diseases (IRDs). It is estimated to affect 1 in 3,500 to 1 in 4,000 people in the United States and Europe, respectively. Mutations in the CNGA1 gene, encoding a subunit of CNG channels in rod photoreceptors, are reported to cause approximately 2% – 8% of autosomal recessive retinitis pigmentosa (arRP). About ViGeneron GmbH ViGeneron is dedicated to bringing gene therapy innovations to people in need. The company is advancing its proprietary gene therapy pipeline to treat ophthalmic diseases, while partnering with leading biopharmaceutical players in retinal diseases, CNS, cardiovascular and other disease areas. ViGeneron’s three novel next-generation gene therapy platforms are geared towards addressing the limitations of existing adeno-associated virus (AAV)-based gene therapies. The first, vgAAV vector platform, enables a superior transduction efficiency of target cells and is designed to overcome biological barriers, thus enabling novel, less invasive routes of administration such as intravitreal and systemic administration. The second, the REVeRT (REconstitution Via mRNA Trans-splicing) technology platform, allows efficient reconstitution of large genes (>5kb) in any tissue which can be targeted with a given capsid. The third, AAV Transactivation is a CRISPR-Cas–based AAV gene therapy platform that enables to regulate one or multiple genes in vivo by increasing or inhibiting their expression. Privately-owned ViGeneron was founded in 2017 by a seasoned team with in-depth experience in AAV vector technology and clinical ophthalmic gene therapy programs and is located in Munich, Germany. For further information, please visit www.vigeneron.com . SOURCE: ViGeneron